This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for Caelyx Biweekly in Heavily Pretreated Patients With Relapsed Ovarian Cancer (NCT00170573), a na phase 2 study. The primary outcome is Occurrence of Palmar-plantar Erythrodysesthesia (PPE).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00170573 |
| Phase | Phase 2 |
| Condition | Ovarian Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 77 |
| Arms in the primary outcome | n/a |
| Lead sponsor | North Eastern German Society of Gynaecological Oncology |
| Results posted | Yes |
2. Clinical Question
Population
Patients with ovarian cancer are identified in the registry record. The official title refers to patients with platinum-sensitive recurrent ovarian, fallopian tube and peritoneal cancer, while the brief title describes heavily pretreated patients with relapsed ovarian cancer. The record does not give further eligibility details or a more specific description of the enrolled population.
Intervention
The registered intervention is Caelyx, described as 40 mg/m² biweekly. The brief summary identifies the schedule’s effect on dose-limiting PPE toxicity and quality of life as areas of interest. The registry record does not provide further treatment-delivery details.
Comparator
No comparator group is described in the registered design. Although the official title refers to a comparison with standard platinum-based therapy, the record identifies a single-group model and does not report a platinum-based group or a like-for-like control comparison.
Primary question
The primary outcome is the occurrence of palmar-plantar erythrodysesthesia (PPE), counted as participants with reported skin toxicity by Common Toxicity Criteria grade. The record describes measurement through study completion, an average of 18 months. It does not report the observed grade-specific counts or an analysis comparing treatment groups.
Read as registered, the primary question is descriptive: what occurrence of PPE was recorded among participants receiving the biweekly Caelyx regimen? The stated focus on quality of life appears in the study title and brief summary, but the registry record does not give a quality-of-life measure or its results. Overall survival is listed as a secondary outcome, but no analysis results are reported. Consequently, the record does not establish a comparative treatment effect for PPE, quality of life or survival.
3. Trial Design
The record identifies this as a phase 2, single-group study with no masking. Allocation is listed as not applicable. The registered intervention is Caelyx at 40 mg/m² biweekly, and the study is marked completed. Enrollment is recorded as 77. The recorded start was 2001-09 and primary completion was 2009-04.
The single-group designation is important for interpretation. The record does not describe a concurrent control arm, random allocation or masking. The official title refers to comparison with standard platinum-based therapy, but the registered design and intervention details do not show such a comparator. The registry record therefore does not support reading the study as a randomized, controlled comparison. Any difference between this study’s outcomes and outcomes from another group or study would not, by itself, isolate a treatment effect.
The primary outcome is occurrence of PPE, recorded as the number of participants with reported toxicity by grade. Its timeframe is through study completion, described as an average of 18 months. Overall survival is listed as a secondary outcome. The registry record does not give further details about visits, follow-up procedures, treatment discontinuation or how outcome observations were collected.
The enrollment figure and the adverse-event figures refer to different registry entries: enrollment is recorded as 77, while the adverse-event group lists 64 at risk. The record does not explain the relationship between these figures. It does not report the number analyzed for the primary outcome or define an analysis population. These omissions prevent assumptions about why denominators differ or which participants contributed to each outcome.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Overall Study
| Stage | Caelyx |
|---|---|
| STARTED | 77 |
| COMPLETED | 64 |
| NOT COMPLETED | 13 |
Reasons for not completing this period:
| Reason | Caelyx |
|---|---|
| Violation of inclusion criteria | 13 |
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
| Characteristic | Unit | Caelyx |
|---|---|---|
| Participants analysed | 64 | |
| Age, Continuous | years | 59 (38 to 81) |
| Sex: Female, Male: Female | Participants | 64 |
| Sex: Female, Male: Male | Participants | 0 |
| Region of Enrollment: Germany | participants | 64 |
| Classification of tumour (according to FIGO ovarian cancer staging): stage IA | participants | 2 |
| Classification of tumour (according to FIGO ovarian cancer staging): stage IB | participants | 0 |
| Classification of tumour (according to FIGO ovarian cancer staging): stage IC | participants | 1 |
| Classification of tumour (according to FIGO ovarian cancer staging): stage IIA | participants | 8 |
| Classification of tumour (according to FIGO ovarian cancer staging): stage IIB | participants | 2 |
| Classification of tumour (according to FIGO ovarian cancer staging): stage IIC | participants | 4 |
| Classification of tumour (according to FIGO ovarian cancer staging): stage IIIA | participants | 2 |
| Classification of tumour (according to FIGO ovarian cancer staging): stage IIIB | participants | 3 |
| Classification of tumour (according to FIGO ovarian cancer staging): stage IIIC | participants | 34 |
| Classification of tumour (according to FIGO ovarian cancer staging): stage IV | participants | 7 |
| Histological grade: grade 1 | participants | 8 |
| Histological grade: grade 2 | participants | 20 |
| Histological grade: grade 3 | participants | 28 |
| Histological grade: grade 4 | participants | 0 |
| Histological grade: unknown | participants | 8 |
| ECOG performance status: 0 | participants | 20 |
| ECOG performance status: 1 | participants | 34 |
| ECOG performance status: 2 | participants | 10 |
| ECOG performance status: 3 | participants | 0 |
| ECOG performance status: 4 | participants | 0 |
| ECOG performance status: 5 | participants | 0 |
6. Primary Endpoint
The registry lists the primary outcome as Occurrence of Palmar-plantar Erythrodysesthesia (PPE), measured over through study completion, an average of 18 months.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is occurrence of palmar-plantar erythrodysesthesia (PPE), a skin toxicity affecting the hands and feet. The registry describes counting participants with reported PPE at Common Toxicity Criteria grades I, II, III or IV. Grade I is described as tingling or altered sensation; grade II as discomfort holding objects or walking, with painless swelling and redness; grade III as painful redness and swelling, including around the nails; and grade IV as peeling, ulceration, blistering or severe pain.
A participant count gives a direct description of how many people were recorded with the event, while grades distinguish reported severity. In general, toxicity endpoints can help characterize tolerability alongside clinical outcomes. They are most interpretable when the record also supplies the number assessed, the number with each grade, the observation period and the rules for handling incomplete observations.
The registry record specifies the endpoint and its timeframe, described as through study completion with an average of 18 months. It does not report the observed PPE counts, grade-specific results, or the number assessed for this endpoint. Thus, the endpoint definition is available, but its observed frequency and precision cannot be evaluated from the posted results.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The record gives a defined primary outcome but no reported analysis results. It identifies PPE occurrence by grade as the primary endpoint and names overall survival as a secondary outcome. The record also indicates two outcome measures, but lists zero analyses. There are no posted estimates, confidence intervals, test results or outcome counts from which to assess the observed PPE frequency or survival findings.
For a participant-count endpoint, the key descriptive information would ordinarily include the number of participants assessed and the number recorded with PPE, ideally separated by grade. Those figures are not reported in the registry record. Without them, neither the observed frequency nor the distribution of severity can be described. A confidence interval would communicate uncertainty around an estimated event frequency; no such interval is reported, and none can be reconstructed from the information in the record.
The single-group design also limits what an outcome frequency could establish, even if it were posted. Without a concurrent comparator, the record would describe outcomes among participants receiving the regimen, not the difference attributable to treatment relative to another therapy. The official title’s reference to standard platinum-based therapy does not alter this limitation because a comparator group is not described in the registered design.
The adverse-event record reports 4 participants with serious adverse events among 64 at risk. It lists Anemia at 4.7% and Thrombopenia at 1.6% among the top serious terms. These entries describe the registry’s safety reporting; they do not replace the missing primary endpoint results. The record also lists 60 affected in the other adverse-event category among 64 at risk. The category details and event-level breakdown are not reported in the registry record.
Enrollment is recorded as 77, whereas the adverse-event group lists 64 at risk. The reason for the different figures is not reported in the registry record. It would be inappropriate to infer that the difference represents withdrawals, exclusions, missing data or a particular analysis population. The record does not provide the flow of participants into each outcome analysis.
Overall, the posted record supports a description of the intended question, design and selected safety entries, but not an estimate of the primary outcome or a comparative conclusion. No claim about the magnitude, precision or statistical significance of a treatment effect can be drawn from the available outcome information.
9. Statistical Methods Explained
What does a participant count describe?
The primary endpoint is a count of participants with an event, classified by PPE grade. In general, this kind of endpoint summarizes whether each assessed participant was recorded as having the event during the stated observation period. A count is not a rate unless an appropriate time denominator is also used. The registry record describes the observation period as through study completion, an average of 18 months, but does not report person-time or a rate analysis.
Counts by grade can show the severity categories recorded, but interpretation depends on clear definitions and complete outcome reporting. Here, the grade descriptions are included, while the observed counts by grade are not reported in the registry record. The record also does not say whether participants could contribute to more than one grade over time, or how a participant’s grade was selected for reporting. No assumptions about those rules are warranted.
What would an estimate and confidence interval add?
In general, an estimate gives a numerical summary of an outcome, while a confidence interval expresses the statistical uncertainty around that estimate under the method used. A narrow interval indicates greater precision than a wide interval, but it does not address biases caused by study design or incomplete measurement. For a participant-level event outcome, an estimate of event frequency would require the relevant event count and assessed denominator. The record does not report the primary outcome count or denominator, and it contains no confidence interval for PPE.
No inferential method for the primary outcome is reported in the registry record. There is no posted test statistic, p-value or adjusted estimate. It is therefore not possible to evaluate whether the investigators used a particular model, whether an assumption was checked, or how uncertainty was quantified. A statistical method should not be attributed to the study solely because it might be commonly used for this type of outcome.
Why does the single-group design matter?
In general, a single-group study can describe outcomes observed among participants receiving an intervention. It cannot, without a suitable comparison, directly estimate how those outcomes differ from what would have happened under another treatment. Random allocation in a comparative study is one way to balance measured and unmeasured factors on average; this record does not describe random allocation or a comparator group. The title’s wording about a comparison does not supply the missing design details.
For that reason, a reported event frequency would be descriptive rather than a randomized treatment contrast. Differences from external studies could reflect differences in participants, follow-up, outcome definitions or care settings, as well as treatment. The registry record does not report an external comparison or an adjusted analysis intended to address these issues.
What do the planning assumptions mean?
The record describes a planning illustration based on the observed effect, with a two-sided significance level of 0.05, equal allocation between two arms and a normal approximation. It describes the target effect as a stated fraction of the observed effect. These are planning assumptions, not a report of the trial’s own design rationale. The registered study is single-group, so equal allocation between two arms does not describe its registered treatment allocation.
In general, sample-size planning asks how many observations a future study would need under specified assumptions about the effect, variability, significance level and desired power. Such an illustration is conditional on those assumptions. It is not evidence that a study had a particular probability of detecting an effect after the fact. The planning illustration is not observed power, and the record does not report the protocol’s sample-size justification. Because the outcome analyses are absent, the observed effect used for planning cannot be evaluated from the posted results.
For a participant-level binary outcome, a planning method also depends on the assumed event probability and the intended comparison. The record does not give the inputs or calculated planning values in the registry record for interpretation, so no required sample size can be stated. The stated assumptions should not be mistaken for evidence that the completed study met a prespecified target.
What can be inferred from the listed safety counts?
The serious adverse-event entry gives a count of affected participants and an at-risk denominator. Such figures are descriptive. In general, they do not show whether an event was caused by treatment, and they do not establish a comparative safety difference without an appropriate comparator and analysis. The registry record does not report a causal assessment, event timing, exposure-adjusted rates or comparative safety analysis.
The record’s separate listing of other adverse events should also be read according to its own categories. A count in that category does not provide an event-by-event account or clarify whether entries overlap with serious events. The registry record does not report those details. No significance tests or confidence intervals for the adverse-event figures are reported.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists two outcome measures: PPE occurrence as the primary outcome and overall survival as a secondary outcome. It reports zero analyses. No analysis results, hypothesis-testing procedures or multiplicity adjustment are reported in the registry record.
In general, multiplicity arises when multiple outcomes, time points, subgroups or statistical tests are examined. Testing several questions can increase the chance of at least one apparently unusual result under a setting where there is no true effect. A prespecified analysis plan can clarify which outcome is primary, how secondary outcomes are treated and whether any adjustment is used. The registry record identifies a primary outcome but does not report such an analysis plan or a procedure for handling multiple comparisons. It is not possible to determine whether multiplicity was addressed outside the registry record.
Missing-data methods are also not reported in the registry record. The record does not state how incomplete outcome assessments, withdrawals, deaths before assessment or missing follow-up were handled. It also does not report the number assessed for PPE or define the analysis population for that outcome. The difference between enrollment of 77 and the adverse-event entry of 64 at risk is not explained, so it should not be interpreted as a missing-data count or an attrition figure.
In general, missing observations can affect both the estimated event frequency and its precision, especially if the likelihood of missingness is related to participant health or outcome status. Without a reported participant flow, outcome denominators and missing-data approach, the extent and direction of any such effect cannot be assessed. No imputation, sensitivity analysis or complete-case approach should be attributed to the study unless it is reported in the registry record.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| Caelyx | 4 | 64 | 6.2% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Caelyx | 60 of 64 |
Most frequent serious adverse event terms
| Event term | Organ system | Caelyx |
|---|---|---|
| Anemia | Blood and lymphatic system disorders | 3 of 64 |
| Thrombopenia | Blood and lymphatic system disorders | 1 of 64 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Caelyx |
|---|---|---|
| PPE | Skin and subcutaneous tissue disorders | 30 of 64 |
| Leucopenia | Blood and lymphatic system disorders | 26 of 64 |
| Thrombopenia | Blood and lymphatic system disorders | 4 of 64 |
Serious events with the highest arm-level rate: anemia (4.7%), thrombopenia (1.6%).
The serious adverse-event entry lists the Caelyx group with 64 participants at risk and 4 affected. It also lists Anemia at 4.7% and Thrombopenia at 1.6% among the top serious terms. These are registry safety figures; the record does not provide further event-level detail or a comparative safety analysis.
The entry for other adverse events lists 64 at risk and 60 affected. The registry record does not explain the categories or provide an event-by-event breakdown for this tally. The serious and other adverse-event entries should be read as separate reported categories, without assuming how individual events or participants overlap between them.
In general, an adverse-event count describes what was recorded, not necessarily what was caused by treatment. A safety comparison requires an appropriate comparator and clear definitions, follow-up and analysis. This record describes a single-group study and does not report a comparative safety estimate, confidence interval or test. It also does not explain the difference between enrollment of 77 and the 64 participants listed at risk for adverse events. The reason is not reported in the registry record.
12. What the Result Does, and Does Not, Mean
The registry record identifies a phase 2, single-group study of Caelyx at 40 mg/m² biweekly, with PPE occurrence as the primary outcome. It describes PPE grading and an observation period through study completion, an average of 18 months. It also lists overall survival as a secondary outcome and includes selected adverse-event figures.
Those details describe the intended study question and the outcomes recorded in the registry. They do not establish the observed frequency or severity of PPE: the primary outcome counts and analyses are not reported. Nor do they provide an estimate of overall survival or quality of life. Although quality of life appears in the study title and brief summary, a specific measure and its results are not reported in the registry record.
The design does not describe a comparator group, despite comparison language in the official title. Therefore, the record cannot support a treatment-effect contrast against standard platinum-based therapy. A single-group outcome, even if reported, would describe participants receiving the regimen rather than establish what would have occurred under another treatment.
The safety entries provide counts and listed serious terms, but do not establish causation or comparative safety. They also do not resolve why the adverse-event at-risk count differs from enrollment. No inference about participant withdrawal, eligibility or missing outcomes follows from that difference alone.
In general, a result is most informative when its estimate, denominator, uncertainty and analysis population are clear and when the design supports the intended comparison. Those elements are not available for the primary endpoint in the posted record. Accordingly, the record’s strongest contribution is its description of the planned clinical question, intervention, endpoint and selected safety reporting—not a quantified conclusion about benefit, harm or comparative effectiveness.
13. Why This Trial Matters Statistically
This trial is statistically informative in part because it illustrates the distinction between a study’s stated question and the design details available for evaluating it. The official title describes a comparison with standard platinum-based therapy, whereas the registered model is single-group and the intervention listing contains Caelyx. Without a registered comparator, the record does not support a like-for-like treatment contrast.
The endpoint is defined in clinically interpretable terms: participant counts for PPE, with grades that distinguish reported severity. Yet endpoint definition alone is not a result. Counts, denominators and uncertainty are needed to estimate the outcome and assess its precision. The primary outcome analyses are not reported, so those statistical questions remain unanswered.
The record also makes clear why safety denominators and participant flow matter. Enrollment is 77, while the adverse-event entry lists 64 at risk. Without an explanation, readers cannot determine whether these figures refer to different populations or reporting processes. Treating the difference as attrition or as an analysis denominator would go beyond the record.
Finally, planning assumptions should be separated from trial results. The stated planning illustration uses equal allocation between two arms, while the registered design is single-group. This contrast shows why planning calculations must be interpreted in the context of the design they assume. They do not establish observed power or supply missing outcome analyses.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The central limitation for statistical interpretation is the absence of reported outcome analyses. The record defines PPE occurrence as the primary endpoint, but does not report event counts, grade-specific results, denominators, estimates or uncertainty. Overall survival is listed as a secondary outcome, but its results are not reported either.
The design information is also limited. The record identifies a single-group model, no masking and allocation as not applicable, but does not describe a comparator group. This conflicts with the comparison wording in the official title and prevents interpretation as a controlled comparison based on the registered design. Further details about eligibility, treatment duration, adherence and follow-up procedures are not reported in the registry record.
Participant accounting is unclear. Enrollment is 77, while the adverse-event group lists 64 at risk. The relationship between these figures, the primary-outcome denominator and any analysis population is not reported in the registry record. Missing-data handling and reasons for any differences are not reported, so no explanation should be inferred.
The record lists quality of life as an area of interest in its title and summary but does not identify a specific measure or report quality-of-life results. It lists overall survival as a secondary outcome without analysis results. Multiplicity procedures, the statistical analysis plan and the protocol’s sample-size justification are not reported in the registry record. These gaps limit evaluation of prespecification, precision and the completeness of outcome reporting.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Caelyx Biweekly in Heavily Pretreated Patients With Relapsed Ovarian Cancer — NCT00170573.
- Registry record last updated 2024-12-16; status last verified 2024-11.
- Results first posted 2024-12-16.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
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18. Record Summary
This completed phase 2 study is registered as a single-group study of Caelyx at 40 mg/m² biweekly. Enrollment is recorded as 77. The primary outcome is PPE occurrence by Common Toxicity Criteria grade, measured through study completion, an average of 18 months. Overall survival is listed as a secondary outcome.
The record reports 4 participants with serious adverse events among 64 at risk, with Anemia at 4.7% and Thrombopenia at 1.6% listed among the top serious terms. It also lists 60 affected in the other adverse-event category among 64 at risk. The reason the adverse-event at-risk count differs from enrollment is not reported in the registry record.
No outcome analyses are reported. The primary PPE counts, survival results, quality-of-life results, confidence intervals and comparative treatment estimates are not reported in the registry record. The registered single-group design does not describe a comparator, despite comparison wording in the official title.